Long-Term Follow-up Protocol

Trial ID or NCT#



recruiting iconRECRUITING


The main objective of this prospective, observational, long-term follow-up (LTFU) study is to evaluate the long-term safety profile of the gene therapy products evaluated by Krystal Biotech, Inc. which have a shared backbone of HSV-1, in participants who received at least one dose of investigational product (IP).

Official Title

A Prospective, Observational, Long-Term Follow-Up (LTFU) Study to Evaluate the Long-Term Safety of the Krystal Biotech, Inc. Gene Therapy Backbone Products Using HSV-1 Backbone

Eligibility Criteria

Ages Eligible for Study: Older than 6 Months
Sexes Eligible for Study: All
Accepts Healthy Volunteers: No
Inclusion Criteria:
  1. - All adult and pediatric participants who received at least one gene therapy treatment in a previous Krystal Biotech, Inc. sponsored study, and have discontinued or completed the parent treatment protocol (which do not have the LTFU embedded into the parent protocol), as applicable. - Consent/Assent must be obtained by the participants (and, parental/legal representative, when applicable) prior to any study-related data being collected. - Participant is willing and able to adhere to the protocol requirements.
Exclusion Criteria:
  1. - Participants who enroll into a non-Krystal Biotech, Inc. interventional gene therapy clinical trial will be excluded from the study.


M. Peter Marinkovich, MD
M. Peter Marinkovich, MD
Dermatologist, Blistering disease specialist, Psoriasis specialist
Associate Professor of Dermatology

Contact us to find out if this trial is right for you.


Sinem Bagci